Erythropoietin (EPO)+/- Filgrastim (G-CSF) vs. Supportive Therapy Alone for Patients With Myelodysplastic Syndromes

Last updated: June 14, 2023
Sponsor: Eastern Cooperative Oncology Group
Overall Status: Completed

Phase

3

Condition

Anemia

Myelodysplastic Syndromes (Mds)

White Cell Disorders

Treatment

Erythropoietin

Filgrastim

Transfusion

Clinical Study ID

NCT00003138
CDR0000065907
E1996
U10CA021115
  • Ages > 18
  • All Genders

Study Summary

RATIONALE: Erythropoietin and colony-stimulating factors such as filgrastim stimulate the production of blood cells. It is not yet known whether erythropoietin with or without filgrastim is more effective than standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.

PURPOSE: Randomized phase III trial to compare the effectiveness of erythropoietin with or without filgrastim with that of standard blood transfusions in reducing the need for transfusions in patients who have anemia associated with myelodysplastic syndrome.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • At least 18 years of age
  • Diagnosis of a myelodysplastic syndrome
  • Refractory anemia (RA)
  • RA with ringed sideroblasts
  • RA with excess blasts (RAEB). RAEB patients must have a bone marrow blast count ofless than 20% and less than 5% blast forms on peripheral blood
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 3
  • Platelet count greater than 30,000/mm^3 (without platelet transfusions)
  • Hematocrit less than 30% (pretransfusion)
  • Bilirubin less than 3 mg/dL
  • Blood urea nitrogen (BUN) less than 40 mg/dL or Creatinine less than 2.0 mg/dL
  • Prior epoetin alfa allowed provided dosage was less than 30,000 units per week forless than 1 month duration
  • At least 1 month since prior erythropoietin
  • At least 2 months since prior recombinant growth factor
  • At least 2 months since prior chemotherapy for other malignancy or autoimmune disease
  • At least 2 weeks since prior androgen or steroids for treatment of myelodysplasticsyndromes

Exclusion

Exclusion Criteria:

  • RAEB in transformation
  • Chronic myelomonocytic leukemia
  • Splenomegaly greater than 6 cm below the left costal margin or greater than 3 timesnormal size
  • Uncontrolled hypertension
  • Sensitivity to E. coli-derived proteins
  • Sensitivity to epoetin alfa or any of its components (e.g., human albumin)
  • Documented iron deficiency. If marrow iron stain is not available, the transferrinsaturation must be greater than 20% or ferritin greater than 100 ng/dL
  • Active infection or bleeding
  • Other uncontrolled malignancy
  • Pregnant or nursing. Fertile patients must use effective contraception.

Study Design

Total Participants: 118
Treatment Group(s): 3
Primary Treatment: Erythropoietin
Phase: 3
Study Start date:
March 04, 1998
Estimated Completion Date:
May 31, 2014

Study Description

OBJECTIVES:

  • Compare the benefit of erythropoietin vs standard transfusion support in reducing transfusion requirements in patients with myelodysplastic syndromes.

  • Compare the clinical response, disease progression, and survival in patients treated with these regimens.

  • Compare the toxicity of these regimens in these patients.

  • Evaluate whether adding filgrastim (G-CSF) or increasing the erythropoietin dose will reduce the transfusion requirement in patients who do not respond to erythropoietin alone.

  • To compare the benefit of erythropoietin versus supportive care alone on quality of life (QOL) in persons with myelodysplastic syndromes.

OUTLINE: This is a randomized, controlled, multicenter, cross-over study. Patients are stratified according to morphologic subtype (refractory anemia [RA] vs RA with ringed sideroblasts vs RA with excess blasts), transfusion requirement (yes vs no), prior erythropoietin treatment (yes vs no), and erythropoietin level (at least 200 mU/mL vs less than 200 mU/mL). Patients are randomized to one of two treatment arms.

  • Arm I (standard transfusion support): Patients receive red cell and platelet transfusions for symptoms or to maintain hematocrit level of 25% or above. Patients undergo bone marrow aspirate and biopsy at 4 months and then every year until development of acute leukemia or completion of study. Patients with progressive disease may cross over to arm II after at least 4 months on study and up to 1 year from the time of randomization. Patients who cross over receive erythropoietin alone.

  • Arm II (Erythropoietin): Patients receive erythropoietin subcutaneously (SC) or intravenously (IV) daily. Patients undergo bone marrow aspirate and biopsy as in arm I. Treatment continues daily for a maximum of 1 year.

Patients with stable or progressive disease at day 120 receive filgrastim (G-CSF) SC daily or 3 days a week and erythropoietin SC daily for up to 6 months. Patients with no response to G-CSF and lower-dose erythropoietin may proceed to a higher dose of erythropoietin.

Quality of life is assessed at baseline, every 4 months during study, and at study completion.

Patients are followed every 4 months for 2 years, every 6 months for 3 years, and then annually for 5 years.

ACTUAL ACCRUAL: A total of 118 patients were accrued for this study.

Connect with a study center

  • CCOP - Colorado Cancer Research Program, Incorporated

    Denver, Colorado 80224
    United States

    Site Not Available

  • Robert H. Lurie Comprehensive Cancer Center at Northwestern University

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Veterans Affairs Medical Center - Lakeside Chicago

    Chicago, Illinois 60611-4494
    United States

    Site Not Available

  • CCOP - Illinois Oncology Research Association

    Peoria, Illinois 61602
    United States

    Site Not Available

  • CCOP - Carle Cancer Center

    Urbana, Illinois 61801
    United States

    Site Not Available

  • CCOP - Cedar Rapids Oncology Project

    Cedar Rapids, Iowa 52403-1206
    United States

    Site Not Available

  • CCOP - Iowa Oncology Research Association

    Des Moines, Iowa 50309-1016
    United States

    Site Not Available

  • MBCCOP - LSU Health Sciences Center

    New Orleans, Louisiana 70112
    United States

    Site Not Available

  • Tufts - New England Medical Center

    Boston, Massachusetts 02111
    United States

    Site Not Available

  • CCOP - Michigan Cancer Research Consortium

    Ann Arbor, Michigan 48106
    United States

    Site Not Available

  • CCOP - Kalamazoo

    Kalamazoo, Michigan 49007-3731
    United States

    Site Not Available

  • West Michigan Cancer Center

    Kalamazoo, Michigan 49007
    United States

    Site Not Available

  • CCOP - Metro-Minnesota

    Saint Louis Park, Minnesota 55416
    United States

    Site Not Available

  • CCOP - Missouri Valley Cancer Consortium

    Omaha, Nebraska 68106
    United States

    Site Not Available

  • CCOP - Southern Nevada Cancer Research Foundation

    Las Vegas, Nevada 89106
    United States

    Site Not Available

  • Veterans Affairs Medical Center - East Orange

    East Orange, New Jersey 07019
    United States

    Site Not Available

  • CCOP - Northern New Jersey

    Hackensack, New Jersey 07601
    United States

    Site Not Available

  • Cancer Institute of New Jersey

    New Brunswick, New Jersey 08903
    United States

    Site Not Available

  • NYU School of Medicine's Kaplan Comprehensive Cancer Center

    New York, New York 10016
    United States

    Site Not Available

  • CCOP - Merit Care Hospital

    Fargo, North Dakota 58122
    United States

    Site Not Available

  • MetroHealth's Cancer Care Center at MetroHealth Medical Center

    Cleveland, Ohio 44109
    United States

    Site Not Available

  • CCOP - Columbus

    Columbus, Ohio 43206
    United States

    Site Not Available

  • CCOP - Geisinger Clinic and Medical Center

    Danville, Pennsylvania 17822-2001
    United States

    Site Not Available

  • CCOP - Sioux Community Cancer Consortium

    Sioux Falls, South Dakota 57104
    United States

    Site Not Available

  • CCOP - Scott and White Hospital

    Temple, Texas 76508
    United States

    Site Not Available

  • CCOP - St. Vincent Hospital Cancer Center, Green Bay

    Green Bay, Wisconsin 54307-3453
    United States

    Site Not Available

  • CCOP - Marshfield Clinic Research Foundation

    Marshfield, Wisconsin 54449
    United States

    Site Not Available

  • Medical College of Wisconsin Cancer Center

    Milwaukee, Wisconsin 53226-3596
    United States

    Site Not Available

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